Clinical trialNCT03952156TerminatedPhase 1/PHASE2Interventional

Gene Therapy Clinical Study in Adult PKU

SponsorHomology Medicines, IncEnrollment10PAH DeficiencyPhenylketonurias

View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

This is a Phase 1/2, open-label, randomized, concurrently-controlled, dose escalation study to evaluate the safety and efficacy of HMI-102 in adult PKU subjects with PAH deficiency. Participants will receive a single administration of HMI-102 and will be followed for safety and efficacy for 1 year.

clinicaltrials.gov ↗

Investigators

The overall officials registered on this study. Names that resolve to a findmyKOL profile link to it; the rest are shown as registered.

Olaf A Bodamer, M.D.
Boston Children's Hospital
Principal Investigator
clinicaltrials.gov ↗

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

13 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

United States

Kaiser Permanente Los Angeles Medical Center
Los Angeles, California, United States
Children'S Hospital of Orange County
Orange, California, United States
University of South Florida
Tampa, Florida, United States
Emory University Hospital
Atlanta, Georgia, United States
Lurie Children'S Hospital of Chicago
Chicago, Illinois, United States
Boston Children'S Hospital
Boston, Massachusetts, United States
Icahn School of Medicine at Mount Sinai
New York, New York, United States
the University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States
Nationwide Children'S Hospital
Columbus, Ohio, United States
Children'S Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Upmc Children'S Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
University of Texas Southwestern Medical Center
Dallas, Texas, United States
University of Utah
Salt Lake City, Utah, United States

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

No drug or biological interventions are in our data for this study — it may test a procedure, device or behavioral intervention, or the intervention list isn't in our current snapshot. See the registry record ↗. Why? →

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start: 10 Jun 2019
Primary completion: 10 Jan 2023
Completion: 1 Aug 2023
Last update posted: 29 Aug 2023
Registered enrollment: 10
Registry id: NCT03952156

clinicaltrials.gov ↗

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.